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Clinical perspective: Advancing hemophilia treatment through gene therapy approaches.

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Title: Clinical perspective: Advancing hemophilia treatment through gene therapy approaches.
Authors: Thornburg CD; National Institutes of Health, National Heart, Lung, and Blood Institute, Division of Blood Diseases and Resources, Bethesda, MD, USA., Pipe SW; Pediatric Hematology-Oncology, University of Michigan, Ann Arbor, MI, USA., Cantore A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy., Unzu C; DNA and RNA Medicine Division, CIMA Universidad de Navarra, Pamplona, Spain., Jones M; Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA., Miesbach WA; Department of Haemostaseology University Hospital Frankfurt, Frankfurt, Germany. Electronic address: miesbach@em.uni-frankfurt.de.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Jun 04; Vol. 33 (6), pp. 2350-2362. Date of Electronic Publication: 2025 Apr 21.
Publication Type: Journal Article; Review
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Clinical perspective: Advancing hemophilia treatment through gene therapy approaches.
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  Data: <searchLink fieldCode="AU" term="%22Thornburg+CD%22">Thornburg CD</searchLink>; National Institutes of Health, National Heart, Lung, and Blood Institute, Division of Blood Diseases and Resources, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Pipe+SW%22">Pipe SW</searchLink>; Pediatric Hematology-Oncology, University of Michigan, Ann Arbor, MI, USA.<br /><searchLink fieldCode="AU" term="%22Cantore+A%22">Cantore A</searchLink>; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Unzu+C%22">Unzu C</searchLink>; DNA and RNA Medicine Division, CIMA Universidad de Navarra, Pamplona, Spain.<br /><searchLink fieldCode="AU" term="%22Jones+M%22">Jones M</searchLink>; Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA.<br /><searchLink fieldCode="AU" term="%22Miesbach+WA%22">Miesbach WA</searchLink>; Department of Haemostaseology University Hospital Frankfurt, Frankfurt, Germany. Electronic address: miesbach@em.uni-frankfurt.de.
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  Data: <searchLink fieldCode="JN" term="%22100890581%22">Molecular therapy : the journal of the American Society of Gene Therapy</searchLink> [Mol Ther] 2025 Jun 04; Vol. 33 (6), pp. 2350-2362. <i>Date of Electronic Publication: </i>2025 Apr 21.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Cell+Press%22">Cell Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>100890581 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1525-0024 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2215250016%22">15250016 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther <i>Subsets: </i>MEDLINE
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        Value: 10.1016/j.ymthe.2025.04.023
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              Text: 2025 Jun 04
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